Sickle Cell Disease

Active Grant List by Core Faculty

Muktar Aliyu M.D., M.P.H., Dr.P.H.

Primary Prevention of Strokes in Nigerian Children with Sickle Cell Disease NIH/NINDS (R21NS080639)
Sickle cell disease (SCD) is the most common genetic disease in the world. Approximately 150,000 Nigerian children are born each year with SCD, making it the country with the largest burden of sickle cell disease in the world. SCD is the most common cause of stroke in children and results in considerable morbidity in affected children. The current primary prevention approach of regular monthly blood transfusion therapy of children at high risk of stroke (identified by elevated transcranial Doppler measurements) is not feasible in a low income country such as Nigeria due to scarcity of supply, cost, and high rate of blood borne infections. In the United States, hydroxyurea (HU) is standard therapy for adults with SCD and may be a reasonable prevention alternative to regular blood transfusion for treatment of primary stroke in high-risk children. This project is a feasibility study to determine the acceptability of randomization to HU vs. placebo for primary prevention of strokes in Nigerian children with sickle cell anemia (SCA) in preparation for a NIH sponsored multicenter, phase III Trial. We will establish a safety protocol for using HU in a clinical trial setting and complete the necessary preparations for a definitive phase III trial. Nigeria has the largest burden of the disease in the world, but the standard therapy for primary prevention of stroke in these high-risk children (recurrent transfusions) is not readily available. A feasibility trial of hydroxyurea for primary prevention of stroke in sickle cell disease will provide evidence for an alternative approach to recurrent transfusions in a part of the world where safe blood services are not readily accessible.
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Primary prevention of stroke in children with Sickle Cell Disease in Sub-Saharan Africa II NIH (R01NS094041)
This dual-center randomized Phase III clinical trial is a continuation of our successful feasibility trial (NCT01801423; 1R21NS080639-NCE), with a goal of determining the efficacy of moderate (20 mg/kg/day) vs. low dose (10 mg/kg/day) hydroxyurea therapy for primary stroke prevention in children with sickle cell anemia in Ghana and Nigeria.
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Hydroxyurea for Stroke Prevention in Children with Sickle Cell Disease in Sub-Saharan Africa
E.W. “Al” Thrasher Award
Hydroxyurea for Stroke Prevention in Children with Sickle Cell Disease in Sub-Saharan Africa. Our goals are: 1) to assess the efficacy of moderate dose HU therapy for secondary stroke prevention when compared to low dose HU therapy among children with SCA; and 2) to determine whether moderate HU therapy decreases the rate of all-cause hospitalizations when compared to low dose HU therapy.

VUMC Sickle Cell Studies